Neighborhood Watch: New Effort to Up Diversity in Clinical Trials

A Boston-based nonprofit is releasing a new video on its novel approaches to help raise awareness of and participation in clinical trials among underserved urban pockets.

Less than 10 percent of patients in clinical trials come from minority communities, the NIH estimates. 

In an effort to change that, the Center for Information and Study on Clinical Research has been going into minority and low-income neighborhoods in major American cities for the past 15 years, meeting with locals, passing out brochures and answering questions about clinical trials to try to garner interest among these poorly represented populations.

Resident feedback told researchers to purge their pitches of generic language and technical jargon and focus on local needs, center founder Ken Getz says.

Over the past couple of years, the center has taken that advice to heart and come up with innovative approaches designed to enhance trial diversity, Getz tells CenterWatch. The center is now circulating videos of their findings in the hope of gaining exposure in local media and on YouTube to try to spread the word.

“What we’d like to do is get these videos to go viral not just within the industry, but so more patients and their families are seeing them. There’s just such a powerful message,” he says.

Earlier this year, the center rented an RV and toured 10 different neighborhoods in and around Los Angeles. The RV, carrying banners for what the center called “Journey to Better Health,” pulled up at sporting events, block parties — anywhere locals gathered, Getz says.

Last year, the center rented retail space in Newark, N.J., and opened what it called “MT Pharmacy.” It had a spiffy corporate logo but its shelves were empty. When curious passerby asked the “pharmacist” what was going on, they were told that drug stores can’t stock themselves without clinical trials. The pharmacist then asked neighbors what kind of research they’d like to see. Each response earned another box on the shelf. By the week’s end, Getz says, MT Pharmacy was full.

“It’s a great way to bring the education right into the heart of the community,” he says.

But it’s also expensive. The center had to rely on donations from Janssen Pharmaceuticals for the RV and Sanofi Pharmaceuticals for the MT Pharmacy.

The videos can be viewed on the center’s website: http://www.CISCRP.org.

Aussies Set Aside $7M for Rare Disease Clinical Trials

The Australian government has set aside $7.1 million to fund six clinical trials for rare and deadly diseases.

The money will go to four universities searching for treatments for pancreatic cancer, Merkel cell carcinoma, myeloma, myelofirbrosis, glioblastoma and traumatic brain injuries.

The announcement comes nearly a year after a local nonprofit group issued a “report card” that found the government, industry and academics needed to do more to improve outcomes for patients with rare diseases.

The Australian government has budgeted $176 million for rare cancer research over the next five years and another nearly $36 million to study cancer genetics.

Rare cancers account for nearly 52 percent of Australia’s total cancer burden, yet have received barely 8 percent of government funding, the nonprofit group Rare Cancers Australia says.

No Fast Track: Rare Disease Med

The FDA has denied Amicus Therapeutics’ request to fast-track an experimental drug aimed at treating Pompe’s disease even though it acknowledged a treatment is needed as “fast as possible” for this rare, often fatal disorder.

The company met with agency officials to discuss plans for a pivotal clinical trial of experimental med AT-GAA, which it wants to test in at least 100 Pompe disease sufferers.

Regulators acknowledged a treatment is needed “as fast as possible” but said Amicus didn’t provide enough data to support accelerated approval.

According to Amicus, the FDA requested more info on the 19 original patients, natural histories from another 100 patients and data from a cohort of an ongoing Phase I/II clinical trial.

Pompe’s disease affects an estimated one in 40,000 people in which the complex sugar glycogen builds up in heart and skeletal muscles. It can cause paralysis and death.

Amicus says it plans to conduct a pivotal trial next year with 100 patients to see if a 12-month dose of AT-GAA can help them walk for six consecutive minutes.

FDA: Specific Trial Data Guidance

The FDA last week issued guidance on technical specifications and considerations for ANDA sponsors on data standards for certain comparative clinical endpoint bioequivalences and skin adhesion and irritation/sensitization clinical trials.

All study-specific data used in generic product evaluation should be submitted using the standards listed in the FDA Data Standards Catalog, the agency said. The guidance includes detailed specification data for the following clinical trials:

• The adhesion study for transdermal delivery systems (TDS) and topical patches;
• The irritation/sensitization study for TDS and topical patches; and
• The comparative clinical endpoint bioequivalence study that uses the endpoints: lesion count, 100 percent clearance of all actinic keratosis legions, total nasal symptom score, treatment success based on the Physician’s Global Assessment and Psoriasis Area Severity Index, intraocular pressure, and therapeutic cure based on clinical and mycological cures.

The guidance includes sample data tables for the Clinical Data Interchange Standards Consortium (CDISC) analysis data model (ADaM). It also offers more information on the agency’s separate guidance titled Providing Regulatory Submissions in Electronic Format – Standardized Study Data (eStudy Data).

Read the guidance here.

Promising Hepatitis B Trial

A German company is claiming early victory for a hepatitis B drug after a Phase I trial showed that the treatment was safe, tolerable and potentially effective.

AiCuris Anti-infective Cures GmbH gave 32 patients either single, random IV doses of its drug, AIC649, or a placebo and monitored the group over the next 84 days. The company says the drug was safe and well-tolerated.

Trial participants also saw jumps in their cytokines, suggesting that AIC649 boosted their immune systems, too, AiCuris says.

A hep B vaccine is available and prevents 95 percent of cases, but the World Health Organization estimates some 257 million people globally are infected with it. In 2015, nearly 900,000 people died from the disease.